Role of Prion Protein in Human Hematopoietic Stem Cells - Full Text View

Sponsors and Collaborators:	University of Utah National Institutes of Health (NIH)
Information provided by:	University of Utah
ClinicalTrials.gov Identifier:	NCT00721864

Purpose

We propose to determine the function of Prion Protein (PrP) in human hematopoietic stem cells. We will use as a model system that provided by nature in PNH patients who have a clone of PrP-null cells and a normal population of PrP+ cells. A competitive engraftment experiment using human PrP-null and Prp+ cells will be performed in a nude mouse followed by engraftment analysis and serial transplantation to assess long term HSC repopulation potential. The contribution of PrP to human LT-HSC proliferation/differentiation will be determined using the cobblestone area forming cell assay.

Condition
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Genetics Home Reference related topics: paroxysmal nocturnal hemoglobinuria

U.S. FDA Resources

Study Type:	Observational
Study Design:	Cohort, Prospective
Official Title:	Role of Prion Protein in Human Hematopoietic Stem Cells

Further study details as provided by University of Utah:

Primary Outcome Measures:

Determine if Prion Protein functions in long term hematopoietic stem cell renewal or proliferation in humans. [ Time Frame: After sample is obtained ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Reconfirm the decreased Prion Protein expression in the peripheral blood and bone marrow of PNH patients [ Time Frame: After sample is obtained ] [ Designated as safety issue: No ]

Biospecimen Retention: Samples With DNA

Biospecimen Description:

Whole Blood

Estimated Enrollment:	20
Study Start Date:	May 2006
Estimated Study Completion Date:	March 2009
Estimated Primary Completion Date:	March 2009 (Final data collection date for primary outcome measure)

Groups/Cohorts
Affected Population Subjects suspected of having Paroxysmal Nocturnal Hemoglobinuria (PNH)

Show Detailed Description

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Patients with paroxysmal nocturnal hemoglobinuria (PNH)

Criteria

Inclusion Criteria:

Diagnosed with paroxysmal nocturnal hemoglobinuria
1. >5% CD55- granulocytes
2. >5% CD59- granulocytes
Able to sign informed consent form.
Age > 18

Exclusion Criteria:

1. Pregnant Women

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00721864

Contacts

Contact: Josef T Prchal, MD	801-581-4220	josef.prchal@hsc.utah.edu
Contact: Kim Hickman, BS	801-581-3707	kimberly.hickman@hsc.utah.edu

Locations

United States, Utah
University of Utah	Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Josef T Prchal, MD 801-581-4220 josef.prchal@hsc.utah.edu
Contact: Kim Hickman, BS 801-581-3707 kimberly.hickman@hsc.utah.edu
Principal Investigator: Josef T Prchal, MD
Sub-Investigator: Charles Parker, MD
Sub-Investigator: Gerald Spangrude, PhD

Sponsors and Collaborators

University of Utah

National Institutes of Health (NIH)

Investigators

Principal Investigator:

Josef T Prchal, MD

University of Utah

More Information

Publications:

Dürig J, Giese A, Schmücker U, Kretzschmar HA, Dührsen U. Decreased prion protein expression in human peripheral blood leucocytes from patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2001 Mar;112(3):658-62.

Risitano AM, Holada K, Chen G, Simak J, Vostal JG, Young NS, Maciejewski JP. CD34+ cells from paroxysmal nocturnal hemoglobinuria (PNH) patients are deficient in surface expression of cellular prion protein (PrPc). Exp Hematol. 2003 Jan;31(1):65-72.

Zhang CC, Steele AD, Lindquist S, Lodish HF. Prion protein is expressed on long-term repopulating hematopoietic stem cells and is important for their self-renewal. Proc Natl Acad Sci U S A. 2006 Feb 14;103(7):2184-9. Epub 2006 Feb 7.

Responsible Party:	University of Utah ( Josef T. Prchal, MD )
Study ID Numbers:	17790, R01HL5077-12
Study First Received:	July 23, 2008
Last Updated:	July 23, 2008
ClinicalTrials.gov Identifier:	NCT00721864
Health Authority:	United States: Institutional Review Board

Keywords provided by University of Utah:

Paroxysmal Nocturnal Hemoglobinuria
Prion Protein
Hematopoietic stem cells
Clonal disorder

Study placed in the following topic categories:

Myelodysplastic syndromes
Paroxysmal nocturnal hemoglobinuria
Urination Disorders
Hematologic Diseases
Myelodysplasia
Myelodysplastic Syndromes
Anemia
Anemia, Hemolytic

Hemoglobinuria
Signs and Symptoms
Proteinuria
Preleukemia
Urologic Diseases
Marchiafava-Micheli disease
Hemoglobinuria, Paroxysmal
Bone Marrow Diseases

Additional relevant MeSH terms:

Urological Manifestations

ClinicalTrials.gov processed this record on January 16, 2009